Proteinuria

Travere Therapeutics and CSL Vifor Announce European Commission Approves FILSPARI® (sparsentan) for the treatment of IgA Nephropathy

Retrieved on: 
Wednesday, April 24, 2024
Nephrology, Kidney disease, Kidney, Div, Proteinuria, European Commission, Kidney failure, CHMP, Irbesartan, Immunoglobulin A, European, EGFR, Patient, CSL Vifor, CSL, Committee, Civil service commission, RAAS, Teaching hospital, Head, Therapy, CMA, SAN, Pharmaceutical industry

SAN DIEGO, April 24, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) and CSL Vifor today announced that the European Commission has granted conditional marketing authorization (CMA) for FILSPARI (sparsentan) for the treatment of adults with primary IgA nephropathy (IgAN) with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). The CMA is granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway.

Key Points: 
  • The CMA is granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway.
  • “The approval of this innovative treatment is based on data from the only head-to-head phase 3 clinical trial in IgAN.
  • CSL Vifor expects to launch FILSPARI in the first European markets in the second half of 2024.
  • In 2021, Travere Therapeutics granted CSL Vifor exclusive commercialization rights for FILSPARI in Europe, Australia and New Zealand.

Eloxx Pharmaceuticals Provides ELX-02 and ZKN-013 Program Updates

Retrieved on: 
Tuesday, April 16, 2024
FDA, Genetics, Drug discovery, ADPKD, Mutation, IPS, Food, Prevalence, Proteinuria, Organoid, OTC, Kidney, Gene, Orphan, RNA, Partnership, UPCR, Almirall, U.S. FDA, FAP, IND, RDEB, Disease, SAD, Patient, Biopsy, Clinical trial, Polycystic kidney disease, ODD, Food and Drug Administration, PKD, Pharmaceutical industry

WATERTOWN, Mass., April 16, 2024 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today provided program updates for ELX-02 and ZKN-013, including Orphan Drug Designation (ODD) for ELX-02.

Key Points: 
  • Eloxx has significantly advanced the development of ELX-02 for the treatment of Alport syndrome with Nonsense Mutations (NMAS).
  • “The recent ELX-02 program updates, including Orphan Drug Designation for ELX-02 for the treatment of Alport Syndrome highlights the significant unmet medical needs of Alport Syndrome patients with Nonsense Mutations,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.
  • The designation was based on a review of the prevalence of NMAS and the data from the Proof-of-concept Phase 2 Study that Eloxx announced top-line results for in 2023.
  • In March 2024, Eloxx announced an exclusive license agreement with Almirall to develop and commercialize ZKN-013 in orphan indications including RDEB and FAP with nonsense mutations.

The U.S. Food & Drug Administration (FDA) Approves Updated LUPKYNIS® (voclosporin) Label to include Long-Term Data from the AURORA Clinical Program

Retrieved on: 
Tuesday, April 30, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Clinical Trials, Other Health, Language, Safety, Woman, Proteinuria, SCRR, EGFR, Patient, Physician, Breastfeeding, Clinical trial, Breast milk, MMF, Lactation, Pharmaceutical industry

The updated label no longer includes language indicating that the safety and efficacy of LUPKYNIS has not been established beyond one year.

Key Points: 
  • The updated label no longer includes language indicating that the safety and efficacy of LUPKYNIS has not been established beyond one year.
  • The updated LUPKYNIS label supports quarterly monitoring of eGFR after the first year of treatment, which is more consistent with current standard clinical care.
  • The safety profile of LUPKYNIS in the updated label remains unchanged and is aligned with the safety findings in the AURORA Clinical Program.
  • The updated label also provides additional lactation data on the transfer of LUPKYNIS to breast milk based on a twice daily dosing regimen.

Seismic Therapeutic Presents Preclinical Data on S-1117, its Novel Pan-IgG Protease Addressing Multiple Pathogenic Mechanisms in Autoimmune Disease, at American Academy of Neurology (AAN) 2024

Retrieved on: 
Friday, April 12, 2024
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, American Academy, Immunoglobulin G, BCR, Tissue, Proteinuria, CDC, Novel, Myasthenia gravis, Doctor of Philosophy, Pharmacology, ADCC, Patient, Blood urea nitrogen, AAN, Polypharmacology, Autoimmunity, Vaccine

Seismic Therapeutic, Inc ., the machine learning immunology company, today announced the presentation of preclinical data for its pan‑immunoglobulin G (IgG) sculpting enzyme candidate, S-1117, at the American Academy of Neurology (AAN) 2024 annual meeting in Denver.

Key Points: 
  • Seismic Therapeutic, Inc ., the machine learning immunology company, today announced the presentation of preclinical data for its pan‑immunoglobulin G (IgG) sculpting enzyme candidate, S-1117, at the American Academy of Neurology (AAN) 2024 annual meeting in Denver.
  • The poster presentation titled, “Preclinical Pharmacology of S-1117, a Novel Engineered Fc-fused IgG Cleaving Enzyme, for Chronic Treatment of Autoantibody-mediated Diseases,” features preclinical in vitro and in vivo results supporting the differentiated therapeutic profile of S-1117.
  • S-1117 reduced IgG effector functions, significantly reducing antibody-dependent cytotoxicity (ADCC) and complement-dependent cytotoxicity (CDC) of peripheral blood mononuclear cells (PBMCs).
  • A murine nephritis model showed reduced complement and immune complex deposition, as well as reduced proteinuria, blood urea nitrogen and renal pathology.

Alpine Immune Sciences Shares Updated Clinical Data from Povetacicept in IgA Nephropathy

Retrieved on: 
Wednesday, April 10, 2024
Health, Other Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, FDA, Congress, Immunoglobulin G, ALPN, University, Glomerular filtration rate, Proteinuria, Survival, Cytokine, MD, Hematuria, UPCR, Webcast, Immunoglobulin A, EGFR, Disease, Nephrology, Patient, WCN, Alpine, Inflammation, Multimedia, Infection, Biomarker, Exhibition, University of Leicester, Autoimmunity, BAFF, Pharmaceutical industry

Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, today shared updated clinical data for povetacicept in IgA nephropathy (IgAN) which will be presented as a late breaking poster at the World Congress of Nephrology (WCN) April 13-16, 2024 in Buenos Aires, Argentina.

Key Points: 
  • Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, today shared updated clinical data for povetacicept in IgA nephropathy (IgAN) which will be presented as a late breaking poster at the World Congress of Nephrology (WCN) April 13-16, 2024 in Buenos Aires, Argentina.
  • RUBY-3 is a multiple ascending dose, multi-cohort, open label, phase 1b/2a study of povetacicept in autoimmune glomerulonephritis, including IgA nephropathy, where povetacicept is administered subcutaneously (SC) once every four weeks.
  • As of March 01, 2024, 41 patients with IgAN had received povetacicept 80 or 240 mg subcutaneously every 4 weeks.
  • These data strongly support the inhibition of APRIL/BAFF pathways by povetacicept and its efficacy in the treatment of IgAN as well as the need for further clinical development.

CSL Vifor and Travere Therapeutics Announce European Commission approves FILSPARI® (sparsentan) for the treatment of IgA Nephropathy

Retrieved on: 
Wednesday, April 24, 2024
Nephrology, Kidney disease, PROTECT, Proteinuria, European Commission, Kidney failure, Professor, CHMP, Irbesartan, European, EGFR, Senior, Patient, Kidney, CSL Vifor, CSL, Medicine, Committee, Civil service commission, RAAS, Teaching hospital, Clinical trial, Therapy, CMA, SAN, Pharmaceutical industry

ST. GALLEN, Switzerland and SAN DIEGO, April 24, 2024 /PRNewswire/ -- CSL Vifor and Travere Therapeutics, Inc., (NASDAQ: TVTX) today announced that the European Commission has granted conditional marketing authorization (CMA) for FILSPARI (sparsentan) for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). The CMA is granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway.

Key Points: 
  • The CMA is granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway.
  • Nephrology and Clinical Immunology at the University Hospital, RWTH Aachen, Germany, and steering committee member for the PROTECT clinical trial.
  • "The approval of this innovative treatment is based on data from the only head-to-head phase-III clinical trial in IgAN.
  • CSL Vifor expects to launch FILSPARI in the first European markets in the second half of 2024.

National Organization for Rare Disorders (NORD) Announces 2024 Rare Impact Award Honorees, Emmy Award-Winning Journalist, Peter Alexander to Host

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Monday, April 22, 2024
Education, FDA, Haemophilia, Risk, Genetics, Policy, Legislation, Louisiana House of Representatives, Diagnosis, Community, Enzyme replacement therapy, DSM-IV codes, Universal Pictures, Usher syndrome, NBC News, Primary immunodeficiency, Metabolic disorder, Therapy, Abbey, Coagulopathy, IgA nephropathy, Hospital, Rare disease, BioMarin Pharmaceutical, Proteinuria, Alpha-mannosidosis, Regeneron Pharmaceuticals, House, Immunoglobulin A, Medicine, Biogen, Universal Studios Hollywood, HFSC, PDT, APDS, Friedreich's ataxia, Disease, Science, Patient, Caregiver, Representative, Activated, Pharmaceutical industry

"Through science and advocacy, they are transforming the lives of those impacted by rare diseases, paving the way for new possibilities in treatment and care."

Key Points: 
  • "Through science and advocacy, they are transforming the lives of those impacted by rare diseases, paving the way for new possibilities in treatment and care."
  • "Having witnessed my sister's journey, I know the challenges rare disease families face are not just physical but also emotional, mental, and financial.
  • In addition to the individual award recipients, the Rare Impact Awards are also honoring companies that have developed treatments changing the lives of those with rare diseases.
  • Regeneron Pharmaceuticals for Veopoz™: The first and only treatment indicated specifically for CHAPLE disease, a rare hereditary immune disease.

Alport Syndrome Market to Witness Upsurge in Growth at a Massive CAGR of 69% by 2034 | DelveInsight

Retrieved on: 
Wednesday, April 17, 2024
USD, Urine, ACE, Finerenone, ESRD, Kidney, Otorhinolaryngology, Nephrotoxicity, Mutation, Hearing, Diagnosis, Glomerular filtration rate, Hearing loss, Blood, Swelling, Proteinuria, Kidney failure, Cochlea, Alport syndrome, Nephrology, EU4, GFR, ADAS, Heredity, Research, Genetic counseling, Kidney disease, Novartis, ARAS, Ophthalmology, COL4A4, Patient, Kidney transplantation, LAS, Dialysis, DSM-IV codes, COL4A3, Quality of life, Genetic disorder, Gene, Pharmaceutical industry

LAS VEGAS, April 17, 2024 /PRNewswire/ -- DelveInsight's Alport Syndrome Market Insights report includes a comprehensive understanding of current treatment practices, Alport syndrome emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Key Points: 
  • Leading Alport syndrome companies such as Eloxx Pharmaceuticals, Chinook Therapeutics (A Novartis company), Bayer, Calliditas Therapeutics, Evotec, and others are developing novel Alport syndrome drugs that can be available in the Alport syndrome market in the coming years.
  • The disorder manifests in different forms such as autosomal recessive Alport syndrome (ARAS), X-linked Alport syndrome (XLAS), and autosomal dominant Alport syndrome (ADAS).
  • The X-linked Alport syndrome (XLAS) is the most prevalent subtype of Alport syndrome with around 11K cases in 2023 in the US while autosomal dominant Alport syndrome (ADAS) is the least prevalent subtype of Alport syndrome.
  • To know more about Alport syndrome treatment guidelines, visit @ Alport Syndrome Management

Alport Syndrome Market to Witness Upsurge in Growth at a Massive CAGR of 69% by 2034 | DelveInsight

Retrieved on: 
Wednesday, April 17, 2024
USD, Urine, ACE, Finerenone, ESRD, Kidney, Otorhinolaryngology, Nephrotoxicity, Mutation, Hearing, Diagnosis, Glomerular filtration rate, Hearing loss, Blood, Swelling, Proteinuria, Kidney failure, Cochlea, Alport syndrome, Nephrology, EU4, GFR, ADAS, Heredity, Research, Genetic counseling, Kidney disease, Novartis, ARAS, Ophthalmology, COL4A4, Patient, Kidney transplantation, LAS, Dialysis, DSM-IV codes, COL4A3, Quality of life, Genetic disorder, Gene, Pharmaceutical industry

LAS VEGAS, April 17, 2024 /PRNewswire/ -- DelveInsight's Alport Syndrome Market Insights report includes a comprehensive understanding of current treatment practices, Alport syndrome emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Key Points: 
  • Leading Alport syndrome companies such as Eloxx Pharmaceuticals, Chinook Therapeutics (A Novartis company), Bayer, Calliditas Therapeutics, Evotec, and others are developing novel Alport syndrome drugs that can be available in the Alport syndrome market in the coming years.
  • The disorder manifests in different forms such as autosomal recessive Alport syndrome (ARAS), X-linked Alport syndrome (XLAS), and autosomal dominant Alport syndrome (ADAS).
  • The X-linked Alport syndrome (XLAS) is the most prevalent subtype of Alport syndrome with around 11K cases in 2023 in the US while autosomal dominant Alport syndrome (ADAS) is the least prevalent subtype of Alport syndrome.
  • To know more about Alport syndrome treatment guidelines, visit @ Alport Syndrome Management

Travere Therapeutics to Present Abstracts on FILSPARI® (sparsentan) in IgA Nephropathy at World Congress of Nephrology and the American Nephrology Nurses Association

Retrieved on: 
Wednesday, April 3, 2024
ART, IgA nephropathy, Congress, Quality of life (healthcare), Patient, SAN, Kidney failure, Immunoglobulin A, RAAS, FSGS, PROTECT, Focal segmental glomerulosclerosis, Abstract, Kidney disease, Therapy, WCN, Proteinuria, ANNA, Communications satellite

SAN DIEGO, April 03, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present nine abstracts in rare kidney disease at the World Congress of Nephrology (WCN) in Buenos Aires, Argentina, on April 13-16, 2024, and the American Nephrology Nurses Association (ANNA) National Symposium in Orlando, Florida, on April 14-17, 2024.

Key Points: 
  • SAN DIEGO, April 03, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present nine abstracts in rare kidney disease at the World Congress of Nephrology (WCN) in Buenos Aires, Argentina, on April 13-16, 2024, and the American Nephrology Nurses Association (ANNA) National Symposium in Orlando, Florida, on April 14-17, 2024.
  • At WCN, the Company will present subgroup analyses of the Phase 3 PROTECT Study of FILSPARI® (sparsentan) in IgA nephropathy (IgAN) showing the treatment effect across participants with different levels of baseline proteinuria.
  • At ANNA, the Company will present additional insights from the HONUS trial, including health-related quality of life (HRQoL) data and the humanistic burden experienced by patients with IgAN and focal segmental glomerulosclerosis (FSGS).
  • “The data we are presenting at WCN and ANNA reinforce the wealth of evidence supporting FILSPARI’s profile to become an effective foundational treatment replacing RAAS inhibition, with the potential to reduce the lifetime risk of kidney failure for patients with IgAN,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics.